Clinical Trial Update: Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations [Phase 1]

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Summary

Phase I trial update of an AAV2-based gene therapy delivering RPE65 for Leber congenital amaurosis due to RPE65 mutations; the study remains in active-not-recru…

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Diseases / indications

• Leber Congenital Amaurosis

Drugs mentioned

• AAV2-hRPE65 vector (AAV2-RPE65 gene therapy vector)

Snippet

Sponsor: University of Pennsylvania | Conditions: Amaurosis of Leber, Retinal Diseases | Phase 1 | Status: ACTIVE_NOT_RECRUITING